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Jesse Gelsinger (June 18, 1981 – September 17, 1999) was the first person publicly identified as having died in a clinical trial for gene therapy. Gelsinger suffered from ornithine transcarbamylase deficiency, an X-linked genetic disease of the liver, the symptoms of which include an inability to metabolize ammonia – a byproduct of protein ...
Clinical trials of gene therapy for sickle cell disease were started in 2014. [221] [222] In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the ...
Introduction. Somatic cell nuclear transfer is a technique for cloning in which the nucleus of a somatic cell is transferred to the cytoplasm of an enucleated egg. After the somatic cell transfers, the cytoplasmic factors affect the nucleus to become a zygote. The blastocyst stage is developed by the egg to help create embryonic stem cells from ...
Human genetic enhancement. An illustration of viral vector -mediated gene transfer using an adenovirus as the vector. Human genetic enhancement or human genetic engineering refers to human enhancement by means of a genetic modification. This could be done in order to cure diseases (gene therapy), prevent the possibility of getting a particular ...
Somatic evolution is the accumulation of mutations and epimutations in somatic cells (the cells of a body, as opposed to germ plasm and stem cells) during a lifetime, and the effects of those mutations and epimutations on the fitness of those cells. This evolutionary process has first been shown by the studies of Bert Vogelstein in colon cancer.
Gene therapy is the genetic engineering of humans, generally by replacing defective genes with effective ones. Clinical research using somatic gene therapy has been conducted with several diseases, including X-linked SCID, [93] chronic lymphocytic leukemia (CLL), [94] [95] and Parkinson's disease. [96]
Generalized anxiety disorder (GAD) is a mental and behavioral disorder, [5] specifically an anxiety disorder characterized by excessive, uncontrollable and often irrational worry about events or activities. [6] Worry often interferes with daily functioning, and individuals with GAD are often overly concerned about everyday matters such as ...
Hematopoietic stem cell transplantation. Hematopoietic stem-cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood, in order to replicate inside a patient and produce additional normal blood cells. [1][2][3][4][5][6] HSCT may be ...